Robert Donnell, Durbin, specialist distributor of pharmaceuticals, medical and relief supplies, examines the increasing prevalence of managed access programmes (MAPs), and the challenges faced in getting the most appropriate drug to the patient in time
Although it is possible to organise MAPs in-house, navigating the varying regulations in the most effective manner can be challenging, says Donnell
Taking a product through clinical trials is a costly process. A report from the Tufts Centre estimated the cost of developing a new prescription medicine and gaining market approval at $2,558 million – a process that could take more than a decade to complete1. Recently there has been an increase in clinical trials involving orphan drugs, designed to meet the need of a rare medical disease. Studies suggest there are 6,000 – 8,000 orphan diseases, of which only 500 have treatment options2.
Orphan drug clinical trials are usually smaller, shorter and successful treatments receive regulatory approval much quicker than medicines for more common diseases. As a result, orphan drug development and regulatory approval is usually a smaller burden on financial and time resources, and has picked up considerable pace.
Product lifecycle
Patients with orphan diseases, and their physicians, tend to be highly active in finding potential treatments, and often investigational drugs are a viable option. Managed access programmes (MAPs) are a blanket term to cover the supply and distribution of these pre-registration and unavailable drugs, and take into account the patient’s needs, local regulations and importation protocols, which vary from country to country.
A MAP can offer a patient access to a drug at various stages of the product lifecycle, including:
- Drugs in clinical development, which have typically completed phase 3 clinical trials, but have not been commercially launched yet
- Drugs that will never be approved, because they treat such a small subset of patients and are therefore not financially viable to develop, but do treat a condition with no other treatment options
- Drugs that have had authorisation in one country but not another. This is common when a company works to achieve approval where the majority of patients reside, but excludes patients living in other areas
- Drugs that have been discontinued, but still retain medical value in a small subset of the population
Finding the right patient
Unlike common diseases, orphan diseases have a much smaller population size, making it challenging to find the number of patients required for a clinical trial. Often specialist physicians are able to identify patient populations quickly and efficiently. However this can dictate the location of the clinical trial, and can result in a need to distribute the orphan drug internationally. This usually warrants the support of a specialist supply and distribution partner, to fulfill the requirement.
If a specialist physician is not available, vast databases of heath insurance and public health databases must be investigated to identify patient hotspots. While there have been many advances in the management of big data, the reality is orphan diseases patient populations are sparse, many are undiagnosed, and identification can be extremely difficult.
Even once identified, there are, and rightly so, stringent criteria in place to ensure the patient is appropriate for the clinical trial. This can be frustrating for patients, particularly when no other treatment option is available.
When to give access
There is an important ethical debate about the supply of unregistered medications to patients whose needs are not met by registered pharmaceuticals. This dilemma is particularly prevalent in considering the situations of the terminally ill or children affected by rare orphan diseases. Emotions can run high and patients, or their physicians can become impatient with regulations, designed to protect their safety. In general, phase 2 data must be present to prove the safety of a drug, before it can be made available through a MAP.
The normal practice is not to supply any medication before full market authorisation and commercialisation. However this should be carefully considered. In the ‘digital age’ patients are connected on an unprecedented level. Forums, patient networks, dedicated disease websites etc. all track and monitor the progress of orphan drugs. As a result, withholding access could be harmful to a company’s reputation and not save lives.
There are many positives to MAPs, a service which can allow pharmaceutical companies to fulfil their mission to help patients, as well as gaining greater market understanding and real patient experience.
Although it is possible to organise MAPs in-house, navigating the varying regulations in the most effective manner can be challenging, and a distraction from core activities. Many companies look to seek the support of a specialist, such as Durbin, to help ensure that the orphan drug reaches commercialisation, whilst most importantly, using MAPs to put the needs of the patient first.
References
- Tufts Center for the Study of Drug Development. Cost to Develop and Win Marketing Approval for a New Drug Is $2.6 Billion. [Internet]. Tufts University. 2014 [cited 2015 Oct 16].
- Ehinger C. Can rare diseases be a viable option for the pharma industry? [Internet]. PMLIVE. 2015 [cited 2015 Sep 18].